Cost-effectiveness analysis of enzalutamide for patients with chemotherapy-naïve metastatic castration-resistant prostate cancer in Japan.

BACKGROUND: We aimed to evaluate cost-effectiveness of enzalutamide in chemotherapy-naïve metastatic castration-resistant prostate cancer patients in Japan. METHODS: A Markov model was developed to capture time spent by patients in various health states: stable, progression and death. Abiraterone acetate and docetaxel were set as active comparators. Clinical outcomes were obtained from the PREVAIL, COU-AA-302 and TAX327 trials. Treatment sequence, concomitant drugs and therapies for adverse events were estimated from responses to a survey by 14 Japanese prostate cancer experts. The analytic perspective was public healthcare payer, with a 10-year time horizon. The incremental cost-effectiveness ratio was estimated from quality-adjusted life-years and Japanese public healthcare costs. Probabilistic sensitivity analysis was performed to assess the robustness of the findings. RESULTS: According to the survey, the most common treatment sequences were (i) enzalutamide â†’ docetaxel â†’ cabazitaxel (enzalutamide-first sequencing), (ii) abiraterone â†’ enzalutamide â†’ docetaxel (abiraterone-first sequencing) and (iii) docetaxel→ enzalutamide â†’ cabazitaxel (docetaxel-first sequencing). In the base-case analysis, enzalutamide-first sequencing saved 1.74 million Japanese Yen versus abiraterone-first sequencing, with a 0.129 quality-adjusted life-year gain (dominant). Enzalutamide-first sequencing had a cost increase of 4.44 million Japanese Yen over docetaxel-first sequencing, with a 0.371 quality-adjusted life-years gain. The incremental cost-effectiveness ratio of enzalutamide-first sequencing versus docetaxel-first sequencing was estimated as 11.94 million Japanese Yen/quality-adjusted life-years. Probabilistic sensitivity analyses demonstrated that, compared with abiraterone-first sequencing, enzalutamide-first sequencing had an 87.4% probability of being dominant. CONCLUSIONS: Results modeled herein suggest that the enzalutamide-first sequencing is more cost-effective than the abiraterone-first sequencing, but less cost-effective than docetaxel-first sequencing for chemotherapy-naïve patients with metastatic castration-resistant prostate cancer.

Are treatment benefits in neuropathic pain reflected in the Self Assessment of Treatment questionnaire?

BACKGROUND/OBJECTIVE: The Self Assessment of Treatment (SAT) questionnaire was developed to reflect key patient reported outcomes of Neuropathic Pain (NP) treatments. This study aimed to understand how patients perceived the relevance and ease of understanding of the questions in the SAT and to recommend modifications based on patient and clinician interviews. METHODS: Semi-structured interviews were conducted with clinicians and NP patients to provide information regarding treatment attributes and the impact of pain. Patients were debriefed on the SAT, a 5-item scale evaluating pain, activity level, quality of life (QoL) and satisfaction with treatment (recommend treatment and undergo treatment again). The SAT has a recall period reflecting back to the start of treatment. The qualitative analysis software ATLAS.ti 5.0 was used to analyze patient transcripts. Changes to the SAT were integrated into the questionnaire for a second round of debriefing interviews. RESULTS: Three NP clinicians and 44 patients (20 painful diabetic neuropathy, 16 HIV-associated neuropathy and 8 post herpetic neuralgia) with a mean age of 60.3 (12.3) years and an even gender distribution were interviewed. Patient treatment experience included anticonvulsants (73%), antidepressants (34%), opioids (25%), and topical medications (41%). Pain descriptors and treatment attributes were similar across the three NP groups. Pain relief was judged the most important treatment attribute, followed by ability to undertake activities. Sleep improvement was another important attribute. Activity limitations and QOL were perceived as too broad and non-specific, and were split into 3 concepts each (activity limitations was split into self care, daily and physical activities and QOL was split into sleep, emotions, and social function). A 7-day recall period was introduced. The item stem and response options were made consistent, and a baseline and follow-up questionnaires were developed (except for the satisfaction items) to enable monitoring onset of treatment benefit and change over time. CONCLUSIONS: The content validity of the revised SAT was improved by the qualitative research, and NP treatment benefits are reflected in a more consistent fashion by the changes. Baseline and follow-up versions make it possible to perform assessments of change over time.

Validation of the Self-Assessment of Treatment Questionnaire among Patients with Postherpetic Neuralgia.

Introduction. A five-item Self-Assessment of Treatment (SAT) was developed to assess improvement and satisfaction with treatment associated with the application of a novel high concentration 8% capsaicin topical patch in clinical trials in patients with postherpetic neuralgia (PHN). This study evaluated the item performance and psychometric properties of the SAT. Methods. The SAT, Brief Pain Inventory, SF-36v2, Short-Form McGill Pain Questionnaire, and Patient and Clinician Global Impression of Change (PGIC; CGIC) scores were measured in two 12-week Phase 3 clinical trials. Factor analysis assessed the underlying factor structure, followed by examination of the reliability and validity of the multi-item domain. Results. Pooled data from 698 patients completing SAT after 12 weeks of treatment were analyzed. A one-factor model combining three of the five items emerged as the optimal solution. Internal consistency reliability of this treatment efficacy factor was high (Cronbach's alpha = 0.89). Construct validity was demonstrated by moderate to high correlations with change in other study endpoints. SAT mean scores consistently discriminated between patient change groups defined by PGIC and CGIC. Conclusions. The measurement properties of the three-item version of SAT are valid and reliable for assessment of treatment with a high concentration capsaicin patch among patients with PHN.

Health economics and outcomes research within drug development: challenges and opportunities for reimbursement and market access within biopharma research.

Healthcare decision makers who determine funding for new medical technologies depend on manufacturers to provide evidence of the technology's efficacy, safety and cost-effectiveness. Constrained budgets and increasing reliance on formal health technology assessment (HTA) have created an abundance of external hurdles that manufacturers must navigate to ensure successful product commercialization. These demands have pushed pharmaceutical companies to adjust their internal structures to coordinate generation of appropriate evidence. In this article we summarize internal and external opportunities for manufacturers to establish a foundation of evidence for successful market access, starting in Phase I of development and continuing throughout the post-approval product lifecycle.

The cost-effectiveness of bimatoprost, latanoprost and timolol in treatment of primary open angle glaucoma in five European countries.

OBJECTIVE: The objective of this cost-effectiveness analysis is to evaluate cost-effectiveness ratios of the intraocular pressure (IOP)-lowering agents bimatoprost, latanoprost and timolol in five major European countries: France, Germany, Italy, Spain and the UK. METHODS: The cost-effectiveness analysis is based on achievement of IOP targets between 13 and 18 mm Hg. Thus, the cost-effectiveness ratios express the costs of having one patient successfully achieving IOP target. The perspective of the analysis is that of the health care sector payer, including costs of medicine and costs of ophthalmologist visits. The time frame is first year of glaucoma treatment. Four treatment strategies are analysed: Timolol as first line with add-on latanoprost or bimatoprost if IOP targets are not met, and latanoprost and bimatoprost as first line with add-on timolol. RESULTS: In the UK, Spain, Italy and Germany the timolol first with add-on of bimatoprost is the least expensive treatment. This strategy dominates both strategies involving latanoprost (as add-on to timolol or as first line) in these four countries. The incremental cost-effectiveness ratio of bimatoprost first-line therapy versus timolol with add-on bimatoprost varies from each country and target (from 305 pounds sterlings to 43,720 euros per patient). In France the timolol first line and latanoprost add-on is not dominated and is the cheapest alternative. The incremental cost-effectiveness ratio of timolol with add-on bimatoprost versus add-on latanoprost lies between 71 euros and 355 euros per patient depending on target (18 and 13 mm Hg, respectively). CONCLUSION: First-line treatment of latanoprost is dominated in all countries. In four out of five countries the timolol first-line therapy with add-on latanoprost is also dominated. Based on this pharmacoeconomic analysis, the most cost-effective strategy seems to be timolol first line with add-on bimatoprost if target is not met after 3 months.

A Markov modelled pharmacoeconomic analysis of bimatoprost 0.03% in the treatment of glaucoma as an alternative to filtration surgery in Italy.

OBJECTIVE: Glaucoma is generally managed by decreasing the intraocular pressure (IOP) to a level believed to prevent further damage to the optic disc and loss of visual field. This may be achieved medically or surgically. The objective of this pharmacoeconomic analysis was to investigate the 4-year costs of bimatoprost 0.03% (Lumigan) eye drops as an alternative to filtration surgery (FS) for glaucoma patients on maximum tolerable medical therapy (MTMT). RESEARCH DESIGN AND METHOD: A Markov model was designed using effectiveness and resource use data from a randomized clinical trial and expert statements (Delphi panel). The RCT covered 83 patients on MTMT. The Model compared bimatoprost with FS. In the bimatoprost model arm patients began treatment with bimatoprost. If target IOP (-20%) was not reached using medical therapy the patient proceeded with FS. In the FS model arm, FS was performed after the first ophthalmologist visit. Unit costs were obtained from an Italian chart and tariffs review (healthcare sector perspective). RESULTS: The RCT showed that 74.7% of the patients delayed the need for FS by 3 months. The Markov model forecasted that 64.2% of the patients could delay the need for FS by 1 year, and forecasted 34.0% could avoid FS after 4 years. The 4-year cost per patient in the bimatoprost and FS arms was E3438 and E4194, respectively (incremental costs of E755). The major cost drivers for the bimatoprost arm were patients who needed combination therapy or FS if the target IOP was not reached. In the FS arm, the major cost drives were the initial surgery costs and pressure-lowering medications used as add-on therapy after FS. CONCLUSIONS: The analysis shows that in a 4-year perspective bimatoprost is cheaper compared to FS. In addition, the postponement of FS associated with bimatoprost may have important implications for waiting list planning.